Bexobrutideg, a first-in-class Bruton’s tyrosine kinase degrader, has been granted orphan drug designation from the FDA in ...

Evaluate, the leading provider of market insights for the pharmaceutical industry, today released its 2025 Orphan Drug Report highlighting forecasts for the orphan drug market out to 2030. The 2025 ...

Pharma market insights provider Evaluate today released its 2025 Orphan Drug Report highlighting forecasts for the orphan drug market out to 2030.

Batoclimab is an anti-FcRn treatment being developed for a range of autoimmune diseases and active thyroid eye disease (TED).

Health Canada has granted approval for Biogen’s Skyclarys (omaveloxolone) to treat Friedreich’s ataxia (FA), a rare, ...

UK-based AstraZeneca’s acquired rare disease drug has met the primary endpoint in a Phase III trial in patients with chronic ...

Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CANF), a biotechnology company developing a pipeline of proprietary ...

House: Johnson & Johnson has received Fast Track designation (FTD) from the U.S. Food and Drug Administration (FDA) for ...

Sapio Sciences, the science-awaretm lab informatics platform, today announced the integration of the NVIDIA BioNeMo platform into the Sapio Lab Informatics Platform. This integration brings AI-driven ...

Completed dosing of first two cohorts in Phase 1/2a REWRITE Clinical Study of KRRO-110 for Alpha-1 Antitrypsin Deficiency (AATD); Interim ...

Privately-held Chinese firm Visirna Therapeutics has announced that a Phase III trial of VSA001 (plozasiran) has met both its primary and all key secondary endpoints. The study focused on patients ...

Alexion Pharmaceuticals’ ravulizumab for the prevention of delayed graft function after renal transplantation was granted FDA orphan ...